Dr. Emil D. Kakkis, M.D., Ph.D. is Chief Executive Officer, President and Founder of Ultragenyx Pharmaceutical. Over the last 25 years, Dr. Kakkis is best known for his work developing novel treatments for rare diseases and advancing the cause of rare disease treatment through advocacy on policy issues by founding and supporting the EveryLife Foundation for Rare Diseases. In 2019, he received BIO’s Henri Termeer Visionary Leadership award for this work and a Lifetime Achievement Award from the National MPS Society.
Dr. Kakkis began his academic research at Harbor-UCLA developing an enzyme replacement therapy for the rare disorder MPS I. After joining BioMarin in 1998, he guided the development and approval of two more treatments for rare diseases, MPS VI and PKU and has contributed to the development of approved or development stage products of four other rare diseases (CLN2, MPS-IVA, PKU, Achondroplasia).
Dr. Kakkis went on to found Ultragenyx in 2010 to focus on developing as many rare and ultra- rare disease therapeutics as possible. The company went public in January 2014 (NASDAQ: RARE) and has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. Since its founding, Ultragenyx has received approvals for XLH, TIO, MPS VII, and LC-FAOD.
Dr. Kakkis graduated from Pomona College, magna cum laude and received combined M.D. and Ph.D. degrees from the UCLA Medical Scientist Program. He completed both a Pediatrics residency and Medical Genetics Training Fellowship at Harbor-UCLA Medical Center.